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Treatment Database

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

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Product Indication Date Designated
Sargramostim To reduce neutropenia and leukopenia and decrease the incidence of death due to infection in patients with acute myelogenous leukemia Mar 06, 1995
Glutamine For use with human growth hormone in the treatment of short bowel syndrome (nutrient malabsorption from the gastrointestinal tract resulting from an inadequate absorptive surface) Mar 06, 1995
Adeno-associated viral-based vector cystic fibrosis gene therapy Cystic fibrosis Feb 15, 1995
Glyceryl trioleate and glyceryl trierucate Adrenoleukodystrophy Feb 14, 1995
Orgotein for injection Familial amyotrophic lateral sclerosis associated with a mutation of the gene (on chromosome 21q) for copper, zinc superoxide dismutase Dec 22, 1994
Coumarin Renal cell carcinoma Dec 22, 1994
Recombinant methionyl brain-derived neurotrophic factor Amyotrophic lateral sclerosis Nov 28, 1994
sodium oxybate Narcolepsy Nov 07, 1994
oxybate Narcolepsy Nov 07, 1994
Tobramycin for inhalation Bronchopulmonary infections of Pseudomonas aeruginosa in cystic fibrosis patients Oct 13, 1994
Coagulation Factor IX (recombinant) Hemophilia B Oct 03, 1994
Aminosidine Visceral leishmaniasis (kala-azar) Sep 09, 1994
Autolymphocyte therapy Renal cell carcinoma Jul 12, 1994
rituximab Non-Hodgkin's B-cell lymphoma Jun 13, 1994
Isobutyramide Sickle cell disease and beta thalassemia May 25, 1994
Ganaxolone Infantile spasms May 25, 1994
Arginine butyrate Sickle cell disease and beta thalassemia May 25, 1994
Treosulfan Ovarian cancer May 16, 1994
Betaine Homocystinuria May 16, 1994
Heme arginate Myelodysplastic syndromes Mar 01, 1994